James Y Garbern Research Project Grant
Awarded to Dr. Paul Tesar – Case Western University
The PMD Foundation sought research grant applications from experienced PMD clinicians and researchers around the world. We received eight outstanding applications offering different areas of study, all valuable to the PMD community. Each application was reviewed by other experts in the field and it fell upon the PMDF Executive Board to choose the one that best met the Foundation’s mission. The project proposed by Dr. Paul Tesar (PhD) and Zachary Nevin (MD/PhD student) of Case Western Reserve University in Cleveland, Ohio was determined to be “the best of the best.” Dr. Tesar describes the concept of their proposed study as follows:
“Stem cells hold great potential to help us understand and treat human diseases. Stem cells possess the ability to turn into any kind of specialized human cell, such as liver cells, heart cells, or nerve cells. Using a technique called “reprogramming,” researchers can convert cells from a sample of a patient’s skin back into “induced pluripotent stem cells”, allowing for the study of cell types that cannot be acquired directly from patients. Pelizaeus-Merzbacher Disease (PMD) is a disorder of myelin-forming oligodendrocytes in the brain and spinal cord. We have obtained samples from patients with a variety of PMD-causing mutations and will generate stem cells and oligodendrocytes from each of these samples in order to study the molecular causes of PMD and myelin damage. We will also use a new gene editing technology to correct the specific mutations in each cell line in order to test the remyelinating potential of corrected cells. This will be the first patient- and disease-specific model of PMD, and we anticipate that it will provide a foundation for future studies into potential drug or stem cell-based therapies to alleviate the life-threatening symptoms of Pelizaeus-Merzbacher Disease..”
To carry out this project, Dr. Tesar’s lab was awarded $50,000 with an additional $50,000 available in 2014 upon demonstration of satisfactory progress. The research was published in 2017: Stem cell-based treatment of Pelizaeus Merzbacher disease.
The PMD Foundation thanks the entire PMD community for making it possible to award this grant, funded by your generosity and fundraising efforts. Your donations and hard work allow us to support promising lines of research that could lead to treatments and an eventual cure.